WO2007089375A3 - Compositions and methods for regulating complement system - Google Patents

Compositions and methods for regulating complement system Download PDF

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Publication number
WO2007089375A3
WO2007089375A3 PCT/US2006/062568 US2006062568W WO2007089375A3 WO 2007089375 A3 WO2007089375 A3 WO 2007089375A3 US 2006062568 W US2006062568 W US 2006062568W WO 2007089375 A3 WO2007089375 A3 WO 2007089375A3
Authority
WO
WIPO (PCT)
Prior art keywords
methods
complement pathway
compositions
complement system
regulating complement
Prior art date
Application number
PCT/US2006/062568
Other languages
French (fr)
Other versions
WO2007089375A2 (en
Inventor
Samuel Jotham Reich
Original Assignee
Acuity Pharmaceuticals Inc
Dejneka Nadine
Samuel Jotham Reich
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority to AU2006337093A priority Critical patent/AU2006337093B2/en
Priority to NZ569368A priority patent/NZ569368A/en
Priority to JP2008547784A priority patent/JP2009521234A/en
Priority to MX2008008302A priority patent/MX2008008302A/en
Priority to CA002634286A priority patent/CA2634286A1/en
Priority to AT06850425T priority patent/ATE466081T1/en
Application filed by Acuity Pharmaceuticals Inc, Dejneka Nadine, Samuel Jotham Reich filed Critical Acuity Pharmaceuticals Inc
Priority to DE602006014026T priority patent/DE602006014026D1/en
Priority to EP06850425A priority patent/EP1966379B1/en
Publication of WO2007089375A2 publication Critical patent/WO2007089375A2/en
Publication of WO2007089375A3 publication Critical patent/WO2007089375A3/en
Priority to IL192282A priority patent/IL192282A/en
Priority to IL223632A priority patent/IL223632A0/en

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Transition And Organic Metals Composition Catalysts For Addition Polymerization (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Lubricants (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

Abstract

The disclosure provided herein provides for methods and compositions for modulating the human complement pathway by reducing the expression or production of one or more complement pathway protein and uses for such methods for treating diseases including ocular disease and macular degeneration related disease. The invention also includes active agents for mediating the modulation of the complement pathway including siRNA designed to cause the RNAi- mediated degradation of complement pathway proteins.
PCT/US2006/062568 2005-12-22 2006-12-22 Compositions and methods for regulating complement system WO2007089375A2 (en)

Priority Applications (10)

Application Number Priority Date Filing Date Title
NZ569368A NZ569368A (en) 2005-12-22 2006-12-22 siRNA compositions and methods for regulating the C3 protein in the complement system
JP2008547784A JP2009521234A (en) 2005-12-22 2006-12-22 Compositions and methods for controlling the complement system
MX2008008302A MX2008008302A (en) 2005-12-22 2006-12-22 Compositions and methods for regulating complement system.
CA002634286A CA2634286A1 (en) 2005-12-22 2006-12-22 Compositions and methods for regulating complement system
AT06850425T ATE466081T1 (en) 2005-12-22 2006-12-22 COMPOSITIONS AND METHODS FOR REGULATING A COMPLEMENT SYSTEM
AU2006337093A AU2006337093B2 (en) 2005-12-22 2006-12-22 Compositions and methods for regulating complement system
DE602006014026T DE602006014026D1 (en) 2005-12-22 2006-12-22 COMPOSITIONS AND METHOD FOR REGULATING A COMPLEMENT SYSTEM
EP06850425A EP1966379B1 (en) 2005-12-22 2006-12-22 Compositions and methods for regulating complement system
IL192282A IL192282A (en) 2005-12-22 2008-06-18 Compositions for regulating complement system
IL223632A IL223632A0 (en) 2005-12-22 2012-12-13 Compositions for regulating complement system

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US75304105P 2005-12-22 2005-12-22
US60/753,041 2005-12-22

Publications (2)

Publication Number Publication Date
WO2007089375A2 WO2007089375A2 (en) 2007-08-09
WO2007089375A3 true WO2007089375A3 (en) 2007-12-06

Family

ID=38234879

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2006/062568 WO2007089375A2 (en) 2005-12-22 2006-12-22 Compositions and methods for regulating complement system

Country Status (11)

Country Link
US (1) US20070178068A1 (en)
EP (2) EP1966379B1 (en)
JP (1) JP2009521234A (en)
AT (1) ATE466081T1 (en)
AU (1) AU2006337093B2 (en)
CA (1) CA2634286A1 (en)
DE (1) DE602006014026D1 (en)
IL (2) IL192282A (en)
MX (1) MX2008008302A (en)
NZ (1) NZ569368A (en)
WO (1) WO2007089375A2 (en)

Families Citing this family (26)

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Publication number Priority date Publication date Assignee Title
JP2006500910A (en) * 2002-04-18 2006-01-12 アキュイティ ファーマシューティカルズ、インク． Means and method for specifically regulating CNS and eye target gene and identification method thereof
US7148342B2 (en) * 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
WO2007095154A2 (en) * 2006-02-10 2007-08-23 The Brigham And Women's Hospital, Inc. Lectin complement pathway assays and related compositions and methods
WO2007133758A1 (en) * 2006-05-15 2007-11-22 Physical Pharmaceutica, Llc Composition and improved method for preparation of small particles
WO2007146953A2 (en) * 2006-06-12 2007-12-21 Exegenics, Inc., D/B/A Opko Health, Inc. Compositions and methods for sirna inhibition of angiogenesis
US7872118B2 (en) * 2006-09-08 2011-01-18 Opko Ophthalmics, Llc siRNA and methods of manufacture
US8580735B2 (en) * 2007-02-05 2013-11-12 Apellis Pharmaceuticals, Inc. Local complement inhibition for treatment of complement-mediated disorders
WO2009108931A2 (en) * 2008-02-28 2009-09-03 Case Western Reserve University Method of treating cancer
PL2320925T3 (en) * 2008-07-10 2016-06-30 Regenesance B V Complement antagonists and uses thereof
US20110229560A1 (en) * 2008-10-06 2011-09-22 Rong-Fu Wang Nlrc5 as a target for immune therapy
US9891219B2 (en) * 2008-10-10 2018-02-13 Mayo Foundation For Medical Education And Research Methods for treating neuromyelitis optica (NMO) by administration of eculizumab to an individual that is aquaporin-4 (AQP4)-IgG autoantibody positive
WO2010065834A1 (en) 2008-12-04 2010-06-10 Opko Ophthalmics, Llc Compositions and methods for selective inhibition of pro-angiogenic vegf isoforms
EP2756845B1 (en) * 2009-04-03 2017-03-15 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of KRAS by asymmetric double-stranded RNA
EA201691215A1 (en) * 2013-12-12 2016-11-30 Элнилэм Фармасьютикалз, Инк. COMPOSITIONS ON THE BASIS OF IRNA TO THE COMPONENT OF THE COMPLEX AND METHODS OF THEIR APPLICATION
US20180085391A1 (en) * 2014-08-08 2018-03-29 Modernatx, Inc. Compositions and methods for the treatment of ophthalmic diseases and conditions
CA2969847A1 (en) 2014-12-10 2016-06-16 Regents Of The University Of Minnesota Genetically modified cells, tissues, and organs for treating disease
EP3405577B8 (en) 2016-01-20 2023-02-15 396419 B.C. Ltd. Compositions and methods for inhibiting factor d
CA3040917A1 (en) * 2016-10-17 2018-04-26 Apellis Pharmaceuticals, Inc. Combination therapy for c3 inhibition
WO2018136827A1 (en) 2017-01-20 2018-07-26 Vitrisa Therapeutics, Inc. Stem-loop compositions and methods for inhibiting factor d
US11866701B2 (en) 2017-11-01 2024-01-09 Alnylam Pharmaceuticals, Inc. Complement component C3 iRNA compositions and methods of use thereof
EP3870711A1 (en) * 2018-10-23 2021-09-01 Gemini Therapeutics Inc. Compositions and methods for treating age-related macular degeneration and other diseases
US11510939B1 (en) 2019-04-19 2022-11-29 Apellis Pharmaceuticals, Inc. RNAs for complement inhibition
BR112022007540A2 (en) * 2019-10-22 2022-07-12 Alnylam Pharmaceuticals Inc COMPONENTS COMPLEMENTARY C3 IRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP4396346A1 (en) 2021-09-02 2024-07-10 Silence Therapeutics GmbH Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell
AU2022378567A1 (en) 2021-10-29 2024-04-11 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
WO2023186056A1 (en) * 2022-04-02 2023-10-05 上海舶望制药有限公司 Composition and method for inhibiting expression of complement component c3 protein

Citations (2)

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WO2003066805A2 (en) * 2001-10-23 2003-08-14 Isis Pharmaceuticals, Inc. Antisense modulation of complement component c3 expression
WO2004009769A2 (en) * 2002-07-24 2004-01-29 The Trustees Of The University Of Pennsylvania COMPOSITIONS AND METHODS FOR siRNA INHIBITION OF ANGIOGENESIS

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WO2004009769A2 (en) * 2002-07-24 2004-01-29 The Trustees Of The University Of Pennsylvania COMPOSITIONS AND METHODS FOR siRNA INHIBITION OF ANGIOGENESIS

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Also Published As

Publication number Publication date
US20070178068A1 (en) 2007-08-02
NZ569368A (en) 2011-11-25
MX2008008302A (en) 2009-01-21
CA2634286A1 (en) 2007-08-09
ATE466081T1 (en) 2010-05-15
AU2006337093B2 (en) 2013-03-14
JP2009521234A (en) 2009-06-04
EP1966379B1 (en) 2010-04-28
EP1966379A2 (en) 2008-09-10
DE602006014026D1 (en) 2010-06-10
EP2221378A1 (en) 2010-08-25
AU2006337093A1 (en) 2007-08-09
IL192282A (en) 2012-12-31
EP2221378B1 (en) 2013-04-17
WO2007089375A2 (en) 2007-08-09
IL223632A0 (en) 2013-02-03
IL192282A0 (en) 2008-12-29

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